Find a Clinical Trial
At OHSU Casey Eye Institute, we are a leader in gene therapy research, bringing help and hope to those facing sight impairment and blindness. Casey is conducting more gene therapy clinical trials than any other institution in the world and is the first to test this groundbreaking approach for several inherited disorders of the retina.
Gene therapy is a treatment that involves replacing non-working genes with healthy copies to slow or halt vision loss. Individuals diagnosed with a genetic eye disease may be a candidate for a clinical trial using gene therapy. We enroll study patients from across the United States and throughout the world.
Clinical trials open to enrollment
These studies are actively seeking new participants. Please call 503-494-0020 or email if you are interested in participating.
Achromatopsia (CNGB3) Gene Therapy Trial
The purpose of this study is to learn about a new gene therapy that may help patients with Achromatopsia. This is the first study that aims to treat Achromatopsia disease by gene therapy. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection into the retina (the lining of the back of the eye that detects light) of one eye. The eye with worse vision will receive the gene therapy.
Achromatopsia (CNGA3) Gene Therapy Trial
The purpose of this study is to learn about a new gene therapy that may help patients with Achromatopsia. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection into the retina (the lining of the back of the eye that detects light) of one eye. The eye with worse vision will receive the gene therapy.
Choroideremia (CHM) Natural History Study
The purpose of this study is to learn more about Choroideremia; how this condition affects a person’s eyes and vision and add to the knowledge base for this rare disease.Leber Congenital Amaurosis (CEP290) Natural History Study
The purpose of this study is to help ophthalmologists and scientists learn about the natural history of CEP290 related retinal degeneration. In medicine, a natural history study is a study that follows a group of people over time who have a specific medical condition or disease.
Stargardt's Macular Degeneration (ABCA4) Gene Therapy Trial
The purpose of this study is to learn about a new gene therapy that may help patients with Stargardt's Macular Degeneration (SMD). This is the first study that aims to treat Stargardt's disease by gene therapy. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection underneath the retina of one eye. The eye with worse vision will receive the gene therapy.
Usher Syndrome Type 1B (MYO7A) Gene Therapy Trial
The purpose of this study is to learn about a new gene therapy being studied in patients with Retinitis Pigmentosa (RP) as a result of Usher Syndrome.This is the first study that aims to treat RP due to Usher Syndrome by gene therapy.The study investigators want to find out if UshStat is safe for use in humans.The gene therapy is given by surgical injection underneath the retina of one eye.The eye with worse vision will receive the gene therapy.
Usher Syndrome Type 2A (USH2A) Natural History Study
The purpose of this research study is to learn more about Usher Syndrome type 2A and Retinitis Pigmentosa caused by specific changes in the USH2A gene and how these conditions affect a person's eyes and vision.
X-Linked Retinitis Pigmentosa (RPGR) Gene Therapy Trial
The purpose of this study is to learn about a new investigational gene therapy that may help patients with XLRP. The study investigators want to find out whether it is safe and to see if it can improve your vision and other symptoms of XLRP. The gene therapy is given by a surgical injection into the retina (the lining of the back of the eye that detects light) of one eye. The eye with worse vision will receive the gene therapy. This study is sponsored by AGTC.
X-linked Retinitis Pigmentosa (RPGR) Natural History Study
X-Linked Retinitis Pigmentosa (RPGR) Natural History Study
The purpose of this study is to learn more about a condition called X-linked retinitis pigmentosa (XLRP). The study investigators want to gain a better understanding of disease progression over time in subjects with XLRP. This study is sponsored by Nightstar.
X-linked Retinoschisis (RS1) Gene Therapy Trial
The purpose of this study is to learn about a new gene therapy that may help patients with X-Linked Retinoschisis (XLRS). This is the first study that aims to treat XLRS disease by gene therapy. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection into the vitreous (a thick, gel-like transparent substance that fills the center of the eye) of one eye. The eye with worse vision will receive the gene therapy.
On-going trials closed to enrollment
These studies are ongoing but no longer seeking new participants.
Achromatopsia (CNGB3) Natural History Study
The purpose of this study is to learn about how Achromatopsia, caused by the CNGB3 gene, affects a person's eyes and vision. Specifically the aim for this natural history study is to learn how this condition affects a patient's eyes and vision over a period of time and what tests will be most helpful for tracking vision changes.
Leber Congenital Amaurosis (RPE65) Gene Therapy Trial
This study is investigating an experimental gene therapy in patients with LCA caused by mutations (changes) in the RPE65 gene.
X-linked Retinoschisis (RS1) Natural History Study
The purpose of this study is to learn more about how to follow changes in vision and other symptoms associated with XLRS. This information is needed for the development of future gene therapy trials.
Completed Trials
These studies are completed.
Ciliary Neurotrophic Factor Device Trial
This study investigated an implant that releases ciliary neurotrophic factor (CNTF), a protein capable of protecting the light-sensitive cells in the eye from further degeneration, in participants with moderate to advanced Retinitis Pigmentosa.
CNTO
This study investigated cell-based therapy in participants with advanced Retinitis Pigmentosa.
Oral Valproic Acid Treatment for Retinitis Pigmentosa
This study investigated the use of oral Valproic Acid (VPA) in participants with autosomal dominant Retinitis Pigmentosa (RP).